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NCT03381729

Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy

Study Summary

The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.

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Interventions

Onasemnogene Abeparvovec-xioiBIOLOGICAL
Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter

Study Locations

FacilityCityStateCountry
UCLALos AngelesCaliforniaUnited States
Stanford UniversityStanfordCaliforniaUnited States
Nemours Children's HospitalOrlandoFloridaUnited States
Ann & Robert H. Lurie Children's HospitalChicagoIllinoisUnited States
Johns HopkinsBaltimoreMarylandUnited States
Boston Children's HospitalBostonMassachusettsUnited States
Washington UniversitySt LouisMissouriUnited States
Nationwide Children's HospitalColumbusOhioUnited States
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvaniaUnited States
UT SouthwesternDallasTexasUnited States
University of UtahSalt Lake CityUtahUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026